NCT00487708已完成2 期
An Open-Label, Phase II Dose Titration Study of ACZ885 (Human Anti-IL-1β Monoclonal Antibody) to Assess the Clinical Efficacy, Safety, Pharmacokinetics and Pharmacodynamics in Patients With NALP3 Mutations
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 34
- 试验地点
- 3
- 主要终点
- Response to treatment and time to relapse after ACZ885 administration according to monthly investigator's clinical assessments, laboratory monitoring, and patient diaries.
研究概览
简要总结
This study will investigate the clinical efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of ACZ885, administered intravenously and subcutaneously to patients with NALP3 mutations whose clinical symptoms are either untreated or insufficiently treated and require medical intervention.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 4 Years 至 75 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients aged 4 to 75 years (inclusive)
- •Body weight ≥ 12 kg and < 100 kg.
- •Females of child-bearing potential must have a negative pregnancy test. Additional birth control details to be provided at screening.
- •Documented molecular diagnosis of NALP3 mutations and clinical symptoms that are either untreated or insufficiently treated and require medical intervention.
- •Patients under anakinra therapy or any other IL-1 blocking therapy, whose clinical symptoms improved under treatment and are willing to discontinue that therapy until a relapse becomes evident.
- •Patients with a very severe characteristics requiring oral prednisone are eligible if the dose is stable (≤ 0.4 mg/kg/day or ≤ 20 mg/day, whichever is lower) for at least 1 week prior to the screening visit. Steroid therapy may be tapered during treatment with ACZ885 at the discretion of the investigator.
- •Parents' or legal guardian's written informed consent (patient's informed consent for ≥ 18 years of age) and child's assent, if appropriate, are required prior to study participation.
排除标准
- •Participation in any clinical trial investigation (except trials with anakinra) within 4 weeks prior to dosing or longer per local regulation
- •Antiinflammatory therapy with colchicine, chlorambucil, dapsone, azathioprine, mycophenolate mofetil, within 3 weeks prior to dosing. Therapeutic antibodies (e.g. anti-TNF-alpha antibodies) must be discontinued at least 60 days before dosing.
- •Donation or loss of 400 mL or more of blood within 8 weeks prior to dosing.
- •A past personal or close family medical history of clinically significant ECG abnormalities or prolonged QT-interval syndrome.
- •History of
- •Immunocompromise, including a positive HIV result.
- •Positive Hepatitis B surface antigen or Hepatitis C test result.
- •Drug or alcohol abuse within the 12 months prior to dosing.
- •Tuberculosis.
- •Renal transplant.
- •Evidence of lymphoma.
- •Active medical condition preventing participation in the study such as infection, poorly controlled diabetes etc.
- •No live vaccinations within 3 months prior to the start of the trial, during the trial, and up to 3 months following the last dose.
研究组 & 干预措施
1
Experimental
ACZ885
干预措施: canakinumab (Drug)
结局指标
主要结局
Response to treatment and time to relapse after ACZ885 administration according to monthly investigator's clinical assessments, laboratory monitoring, and patient diaries.
时间窗: Every month
次要结局
- Assessment of safety,tolerability and immunogenicity of ACZ885 at each clinical visit. Evaluation of ACZ885 PK and PD at each clinical visit Evaluate efficacy towards hearing loss(every 4 months),kidney function (every 4 months),neurological symptoms(Every month)
研究者
研究点 (3)
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